Not currently recruiting at University of California Health

A Phase 2/3 Study in Adult and Pediatric Participants With SCD

a study on Sickle Cell Anemia Anemia

Summary

Eligibility
for people ages 6 months to 65 years (full criteria)
Location
at UCLA
Dates
study started
completion around

Description

Summary

The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.

Official Title

A Phase 2/3 Randomized, Multicenter Study of GBT021601 Administered Orally to Participants With Sickle Cell Disease and an Open-Label Pharmacokinetics Study in Pediatric Participants With Sickle Cell Disease

Details

This is a three-part, multicenter, Phase 2/3 study of orally administered osivelotor in participants with sickle cell disease (SCD).

Part A will evaluate the safety, tolerability, and efficacy of osivelotor in adult participants with SCD to determine an optimal dose.

Part B will evaluate the efficacy of osivelotor versus placebo in adult and adolescent participants with SCD for 48 weeks.

Part C will evaluate the pharmacokinetics (PK) and safety of single and multiple doses (MD) of open-label single arm osivelotor administered to pediatric participants.

Keywords

Sickle Cell Disease, Sickle Cell Anemia, Osivelotor

Eligibility

You can join if…

Open to people ages 6 months to 65 years

Part A, Part B, and Part C:

  • Male or female with SCD
  • Participants with stable Hb value as judged by the Investigator
  • For participants taking hydroxyurea and/or L-glutamine, the dose must be stable for at least 90 days prior to signing the ICF or assent and with no anticipated need for dose adjustments during the study in the opinion of the Investigator.

Part B:

  • Participants with SCD ages 12 to 65 years, inclusive
  • Participants with more than or equal to 2 and ≤ 10 VOCs within 12 months of Screening.

You CAN'T join if...

Part A, Part B, and Part C:

  • Participants who had more than 10 VOC within 12 months of screening
  • Female participant who is breastfeeding or pregnant
  • Participants who receive RBC transfusion therapy regularly or received an RBC transfusion ---for any reason within 90 days of Day 1
  • Participants hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF

Locations

  • Lundquist Institute for Biomedical Innovation at Harbor- UCLA Medical Center not yet accepting patients
    Torrance California 90502 United States
  • McGovern Medical School/Health Science Center Houston accepting new patients
    Houston Texas 77030 United States
  • Memorial Hermann - TMC Investigational Drugs, IDS Pharmacy accepting new patients
    Houston Texas 77030 United States
  • Memorial Hermann Hospital, Texas Medical Center - Clinical Research Unit (CRU) accepting new patients
    Houston Texas 77030 United States
  • UT Physicians Comprehensive Sickle Cell Clinic accepting new patients
    Houston Texas 77030 United States

Details

Status
accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
(estimated)
Sponsor
Pfizer
Links
To obtain contact information for a study center near you, click here.
ID
NCT05431088
Phase
Phase 2/3 research study
Study Type
Interventional
Participants
Expecting 517 study participants
Last Updated